Rare Disease

This technology is a monoclonal antibody which binds annexin for the inhibition of Funsobacterium nucleatum (Fn) invasion and proliferation to treat and prevent Fn related diseases including colorectal cancer and Familial Adenomatous Polyposis (FAP).

This technology is an ATP7B knockout mouse capable of recapitulating the pathophysiology of Wilson disease.

This technology is an enzyme that is involved in synthesizing a precursor to bis(monoacylglycero)phosphate (BMP) and can be used to modulate BMP levels and treat BMP-related disorders.

This technology is a naturally occurring non-coding RNA that regulates the SLC2A1 gene, allowing for therapeutic regulation of Glut1 for treatment of diseases in which Glut1 is under-expressed.

This technology identifies both a biomarker for autoimmune hemolytic anemia (AIHA) and a potential treatment that can prevent AIHA onset (i.

This technology describes a brain-specific OCRL1 mutant transcript that will be further studied to elucidate neuron-specific functions of the gene.

This technology is a knock-in mouse model with a mutated ryanodine receptor to study catecholaminergic polymorphic ventricular tachycardia (CPVT) and other arrhythmias.

This technology is a targeted anti-tumor agent designed to treat adenoid cystic carcinoma (ACC) by utilizing retinoid receptor modulation to promote cancer cell differentiation and selective cell death.